Inhibiting or downregulating glycogen synthase by creating premature stop codons using antisense oligonucleotides
AssigneeGenzyme Corporation
InventorsCarol A. Nelson, Bruce M. Wentworth, Ronald K. Scheule, Timothy E. Weeden, Nicholas P. Clayton
Life of this patent
Estimating the term…
Abstract
The present disclosure relates to antisense oligonucleotides (AONs) for modulating the expression of glycogen synthase. AONs of the present disclosure may be useful in treating diseases associated with the modulation of the expression of the enzyme glycogen synthase, such as Pompe disease. Also provided by the present disclosure are compositions comprising AONs, as well as methods of down regulating mRNA coding for glycogen synthase, methods for reducing glycogen synthase in skeletal and cardiac muscle, and methods for treating Pompe disease.
Claims18
A method of down regulating mRNA coding for glycogen synthase comprising administering an effective amount of an antisense oligonucleotide to an animal, wherein the antisense oligonucleotide comprises SEQ ID NO:58, 59, or 60, and wherein the hybridization of the antisense oligonucleotide to the nucleic acid sequence encoding for glycogen synthase induces exon skipping.
A method for reducing glycogen synthase in skeletal and cardiac muscle comprising administering to an animal an effective amount of an antisense oligonucleotide, wherein the antisense oligonucleotide comprises a sequence selected from the group consisting of SEQ ID NO:58, 59, and 60, and wherein the hybridization of the antisense oligonucleotide to the nucleic acid sequence encoding for glycogen synthase induces exon skipping.
A method for treating Pompe disease comprising administering to an animal an effective amount of an antisense oligonucleotide, wherein the antisense oligonucleotide comprises a sequence selected from the group consisting of SEQ ID NOs:58, 59, and 60, and wherein the hybridization of the antisense oligonucleotide to the nucleic acid sequence encoding for glycogen synthase induces exon skipping.
Dependencies are inferred from the claim text.